As a female Bible college student, an outside marker of pious and godly womanhood consisted of having a male significant other. While this is not strictly a religious sentimentality about…
It's a parent's worst nightmare-- a rare disease that strikes early in life, and without treatment, can cause a patient's death. Tyrosinemia type 1 (TYR1), is a form of a…
According to a story from Charcot Marie Tooth News, a small subdivision of the National Institutes of Health (NIH) is leaning into its role to assist with research into rare…
According to a story from PR Newswire, the pharmaceutical company Therapix Biosciences recently announced the results of a Phase IIa study for its drug candidate THX-110. The therapy is indicated…
According to a story from medpagetoday.com, researchers are beginning to realize that symptoms of pediatric spondyloarthritis present themselves in a distinct way when compared to the symptoms that are experienced…
It is international Pompe Day! “Together we are Strong” Today is meant to help create awareness for the Lysosomal Storage Disorder Pompe Disease, which is both rare and underdiagnosed. Pompe…
According to a story from femalefirst.co.uk, Jas Shaw of the electronic music project Simian Mobile Disco was recently diagnosed with AL amyloidosis. The group was planning a tour of the…
Hope everybody is enjoying the warm weather! This week we have patient stories about a woman with MS who started a support group and a rare mom who took issues…
According to a story from QQ Malaysia Online, patient advocates are looking for ways to raise awareness of rare diseases in Malaysia and get help to forgotten patients that desperately…
According to a story from PR Newswire, a recent survey suggests that a greater number of U.S. citizens know about the existence of pancreatic cancer than ever before. This survey…
According to a story from PR Newswire, the science and tech company Merck recently announced that Mavenclad (cladribine) was recently approved for public use by the UAE Ministry of Health…
Now these are the kind of stories we love to read about! Canadian Revée Agyepong is the first adult in Canada to be cured of sickle cell anemia through stem cell…
According to a story from scimex.org, a recent data review is calling into question the previously held consensus that common pain medicines such as NSAIDs and paracetamol could make inflammatory…
No matter which way you spin it, Ehlers-Danlos syndrome (EDS) is generally not a great time. EDS is a rare genetic condition that affects connective tissue throughout the body. It…
According to a story from ankylosingspondylitisnews.com, a significant proportion of patients stopped taking a biosimilar for infliximab within six months of transitioning to the new medication. This suggests that the…
According to a story from The Irish Times, a new campaign is encouraging people with the rare lung condition idiopathic pulmonary fibrosis to keep exercising. The campaign is centered around…
A recent story from Science Daily is highlighting just how difficult it can be for patients with rare diseases to get an accurate diagnosis. For patients with mitochondrial disease, the…
According to a story from Neurology Advisor, a retired physician experienced the challenges of being a rare disease patient first hand after being diagnosed with primary periodic paralysis thanks to…
According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval from the European Medicines Agency (EMA).…
According to a recent story from CityNews Toronto, Alara Ozkose's Dravet syndrome was preventing her from being able to live a life that was even close to normal. She was…
According to a story from Newswise, researchers at the University of Texas Southwestern Medical Center are reporting the a new drug developed for the treatment of rare, pediatric cancers, has…
According to a story from the Seattle Times, a massive $150 million gift from Richard and Susan Rogel to the University of Michigan is slated to go towards the school's…
According to a story from the Independent, father Nick Sireau is worried that Brexit, the United Kingdom's plan to withdraw from the European Union, could affect the ability of his…
According to an announcement from the pharmaceutical company Retrophin, Inc., the company is starting to move forward with phase 3 trials for its drug candidate sparsentan. The drug is in…
According to a story from 4-traders.com, the pharmaceutical company SteroTherapeutics recently announced that its drug candidate ST-002 was awarded Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).…
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